The company intends to use the funds for the upcoming Phase 3 trial for its lead product candidate NSR-REP1 for choroideremia, an ongoing Phase 1/2 clinical trial of its product candidate NSR-RPGR for X-linked retinitis pigmentosa, and a planned Phase 1/2 clinical trial of a gene therapy product candidate for an inherited form of macular dystrophy.

Founded in 2014 by ophthalmology researchers at the University of Oxford and led by David Fellows, chief executive officer, Nightstar is a retinal gene therapy company focused on developing and commercializing novel, potentially curative one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness, and for which there are no other treatment options.
Its lead program, NSR-REP1, is being developed as a one-time treatment for patients with choroideremia, a rare, inherited cause of progressive blindness that affects approximately one in every 50,000 people. The second gene therapy program, NSR-RPGR, is being evaluated in the first Phase 1/2 gene therapy clinical trial to treat patients with X-linked retinitis pigmentosa.
In addition to these two programs, the company is evaluating several other retinal gene therapy product candidates for entry into clinical trials.